Thalassemia Faq's

The treatment of most cases of thalassemia intermedia involves close monitoring and observation.

• Patients with satisfactory hemoglobin (Hb) levels are frequently monitored.
• These patients usually require blood transfusions only on certain occasions such as the presence of intercurrent infections, hypersplenism, or other illnesses.
• If patients can no longer maintain an Hb level of more than 6 g/dL, they are either started on a regimen of regular blood transfusions or a different option, such as splenectomy, should be tried. Patients with evidence of hypersplenism have a good chance to have their need for blood transfusion reduced or totally eliminated; this might last for months or years. Others may try to administer one of the drugs that may induce stress erythropoiesis and raise Hb levels. Hydroxyurea has been frequently used for this purpose. In separate studies on a large number of patients, a response rate exceeding 75% was reported after long-term therapy. However, this high rate of response was not confirmed by other studies until a study on a small number of patients with β thalassemia major and intermedia treated with hydroxyurea demonstrated a response rate of more than 82%.
  • The initial regimen includes transfusion of 10-15 mL of packed red blood cells (PRBC) every 4-5 weeks to keep the Hb level over 10 g/dL.
  • Blood transfusions should be leukocyte poor to avoid sensitization because such patients have the potential of becoming transfusion dependent in the future.
  • Patients should be checked and typed for minor blood groups to avoid further difficulties in providing appropriate blood for them in the future.
  • Identification of a small group of dedicated donors minimizes the risk of viral exposure and alloimmunization.
• Iron status must be carefully monitored, and patients with iron overload should be treated with an aggressive chelation regimen as soon as indicated.
  • A popular chelation regimen includes administration of deferoxamine 5 days per week as a subcutaneous infusion over 8-12 hours. This regimen revolutionized the treatment of β thalassemia major in patients regularly receiving PRBC transfusions and resulted in longer survival and near-normal quality of life. An oral iron chelator, deferasirox (Exjade), has been in use in the United States for some time now. This agent has a long half-life and, for this reason, is orally administered once daily. Several studies have confirmed the long-term efficacy and safety of this agent. It is now much more popular and, due to the ease and convenience of administration and better compliance, is probably replacing deferoxamine
  • A similar dose is often administered at the time of blood transfusions to help bind the transfused iron (from hemolyzed RBCs).
• Nutritional deficiencies should be addressed and treated.
  • A folic acid supplement should be administered.
  • Vitamin C supplementation has been effective in enhancing the efficiency of chelating iron from tissues.
• Patients who have undergone a splenectomy should be placed on prophylactic antibiotics and be treated empirically for any signs of infection or fever while awaiting the results of blood cultures.
• Appropriate vaccinations, including the polyvalent polysaccharide pneumococcal, the Haemophilus influenzae type b, and the quadrivalent meningococcal vaccines, should be administered to patients 1-2 weeks before splenectomy.
• Patients with severe β thalassemia intermedia are prone to infection with Yersinia enterocolitica, similar to individuals with the severe forms of thalassemia major. For this reason, patients who develop fever without clear cause should receive appropriate treatment even if culture results are negative.
• Young children should have their growth and development closely monitored; any deviation from normal should alert the physician to further investigate the need for blood transfusions.
• Failure to thrive, exercise intolerance, bone deformities and fractures are all potential complications; the health care provider should always look for ways to prevent these complications or at least identify and treat them early with regular blood transfusions, which are frequently effective in reversing or preventing their progress.
• In patients with severe thalassemia intermedia who require aggressive therapy to sustain life, bone marrow transplant, similar to that performed in patients with thalassemia major, is a reasonable alternative to transfusion and chelation if a matched sibling donor is available.
• Many studies have shown that patients with thalassemia intermedia who are not on regular blood transfusion because of their milder symptoms nevertheless develop major complications related to their chronic anemia and ineffective erythropoiesis (IE). Considering the cost-benefit balance of regular treatment in patients with thalassemia major, most patients with thalassemia intermedia would apparently benefit from similar therapy to prevent the complications, rather than waiting to deal with such complications when they occur.

Read Users' Comments (0)